Ignite Creation Date:
2024-05-06 @ 2:18 PM
Last Modification Date:
2024-10-26 @ 1:28 PM
Study NCT ID:
NCT04273243
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-02-06
First Post:
2020-02-14
Brief Title:
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
Sponsor:
Amicus Therapeutics
Organization:
Amicus Therapeutics
Study Overview
Official Title:
Long-Term Follow-Up of AT-GTX-501 scAAV9 Gene Transfer in Subjects With CLN6 Batten Disease
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a long-term safety and efficacy study in subjects with CLN6 Batten disease who previously received a single intrathecal administration of AT-GTX-501
Detailed Description:
This is a long-term safety and efficacy study in subjects with CLN6 Batten disease also know as variant late infantile neuronal ceroid lipofuscinosis associated with mutations in the CLN6 gene vLINCL6 diseasewho previously received a single intrathecal administration of AT-GTX-501 The assessments described in this long-term follow-up LTFU study AT-GTX-501-02 are performed following and in addition to the initial 2 years of post-treatment assessments in the treatment study AT-GTX-501-01 In this LTFU study subjects complete safety and efficacy assessments throughout the studys 3-year duration Combining the duration of the initial treatment study and this LTFU study the overall duration reflects a follow-up period up-to 5 years since gene transfer via AT-GTX-501
The primary outcome for this study is to assess the long-term safety of AT-GTX-501 in subjects with CLN6 Batten disease
The secondary outcome measure of this study is to assess the long-term efficacy of AT-GTX-501 in subjects with CLN6 Batten disease
The primary outcome for this study is to assess the long-term safety of AT-GTX-501 in subjects with CLN6 Batten disease
The secondary outcome measure of this study is to assess the long-term efficacy of AT-GTX-501 in subjects with CLN6 Batten disease
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None